US legislators brought in the new year by narrowly delaying a package of scheduled cuts that some economists postulated would severely dampen the US' economic outlook while subjecting federal agencies to massive budget cuts-an approximately $320 million hit to FDA's budget alone. But while most focused on the legislation's obvious effects on agency budgets, it contained another section that could enormously impact the regulatory profession in the long term.
Section 601(b) of the American Taxpayer Relief Act (ATRA) of 2012, entitled "Advancement of clinical data registries to improve the quality of health care," directs Medicare to accept data on quality measures-required under the Patient Protection and Affordable Care Act of 2010, better known as Obamacare.
That part isn't unusual. What is, observes Peter Orszag, formerly the director of the Congressional Budget Office (CBO), is that quality data can now be submitted to Medicare in the form of patient registries.
"These registries collect information on patient characteristics, patterns of care and outcomes that can be crucial to evaluating what medical techniques and strategies work and which ones don't," wrote Orszag in a Bloombergopinion piece. "Unfortunately, registries are not as widespread as they should be-and the ones that exist often are limited to particular types of care."
The registries have proven to be highly useful to regulators in the past, and in particular for getting the data needed to support clinical trials and subsequent product submission to the US Food and Drug Administration (FDA). The classic case may be that of the Cystic Fibrosis Foundation (CFF), whose database of patients has already been able to support the development of numerous products, including Vertex Pharmaceuticals' Kalydeco (ivacaftor), approved early last year.
As Regulatory Focus wrote in 2012, the interest in patient registries seems to be growing in leaps and bounds in recent years, with numerous government entities showing their interest in using the data obtained by them. The Association of Healthcare Research and Quality (AHRQ), the US government's clearing house for quality data, announced that it would begin a registry of registries to track them all. Other agencies, such as FDA, have expressed their interest in formulating one-off registries to meet unfilled needs, and private companies such as Sanofi have also put money into supporting registries with the potential to support products still in development.
Overall, though, there aren't nearly as many patient registries as some would like. Orszag compares patient registries to a pool of data that is an "inch wide and a mile deep," while insurance data pools are a "mile wide and an inch deep." Marrying the two pools of data-and simply increasing the incentives to provide it in the form of patient registries-should go a long way toward increasing their usefulness and applicability, he opined.
ATRA, for its part, requires Medicare to qualify clinical data registries in accordance to a number of considerations: transparency mechanisms for data elements and measures, submission of data with respect to multiple payers, the submission of timely performance reports and its ability to support quality improvement initiatives for participants.
The registries' ability to drive quality outcomes for Medicare could significantly drive down costs while giving regulators new tools to review the efficacy and safety of approved products. In a presentation given to the Regulatory Affairs Professionals Society's (RAPS) 2011 Horizons conference, Boston Consulting Group consultant Stefan Larsson noted that the use of the tools in EU countries had been instrumental in developing best practices for care and the use of medications.
"By identifying variations in outcomes within the same population, registries make it possible to benchmark and assess comparative performance at the clinic, regional, national, or even international level," Larsson wrote in a prior article. "In-depth analysis of the causes behind variations in performance can lead to the identification of best practices. Active dissemination of those best practices and support to enable their adoption improve outcomes and reduce variations in clinical practice."
The legislation also includes a mandatory study to be done before the data collection program, set to begin in 2014, starts. The Government Accountability Office (GAO) will be required to submit to Congress a study on the plan's potential impact on the quality of the program, its use of health information technology and its ability to drive payment system incentives properly.
Regulatory professionals, meanwhile, could stand to benefit as well, though such an effect might not be positive. Deeper pools of data will likely allow companies to better target medicines to specific patient groups, such as those with a particular gene defect (which is what Kalydeco treats). This might allow for smaller clinical trials-just the thing FDA recently sought to provide insight on in its Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products draft guidance.
By requiring less randomization and fewer patients, regulatory professionals could reduce the costs of a regulatory submission and save money up front.
After the submission of a product, opportunities could become less certain. FDA could one day use the registries to show, for instance, that a product does not work as well as initial data indicated, and potentially restrict its use in various populations. Conversely, if the registries track a drug or device's off-label usage, that data could come to support new indications for use, or even show that a product does not require post-marketing safety restrictions such as a Risk Evaluation and Mitigation Strategy (REMS).
Orzsag agrees with that assessment. "A couple of years ago, a research team from Duke University was able to use the cardiovascular data to discover that more than 20 percent of heart patients who received implantable cardioverter defibrillators should not have received them, based on evidence guidelines. Unnecessary implants not only raise costs but also pose dangers for patients; the study found those with implants who didn't need them stood a risk of in-hospital death and post-procedure complications that was significantly higher than that of patients who fit the evidence guidelines."
"Without a registry, such analyses are not possible," he concluded.